Rhizomelic chondrodysplasia punctata

Summary

Rhizomelic chondrodysplasia punctata (MONDO:0015776) is a disease with 4 cohort genes and 2 clinical trials. The dominant Reactome pathway is Peroxisomal protein import (4 cohort genes). Top therapeutic interventions include ppi-1011.

At a glance

• Prevalence: Unknown (Worldwide) [Orphanet-validated]
• Cohort genes: 4
• ClinVar variants: 130
• Phenotypes (HPO): 20
• Clinical trials: 2

Clinical features

Epidemiology

Prevalence records

2 prevalence record(s), Orphanet:

Type	Class	Value	Geography	Validation
Prevalence at birth	1-9 / 1 000 000	0.7	Europe	Validated
Prevalence at birth	1-9 / 1 000 000	0.5	United States	Validated

Signs & symptoms

Clinical features (HPO)

20 HPO clinical features (Orphanet curated; top 20 by frequency):

HPO ID	Term	Frequency
HP:0000164	Abnormality of the dentition	Very frequent (80-99%)
HP:0000252	Microcephaly	Very frequent (80-99%)
HP:0000286	Epicanthus	Very frequent (80-99%)
HP:0000518	Cataract	Very frequent (80-99%)
HP:0000944	Abnormal metaphysis morphology	Very frequent (80-99%)
HP:0000958	Dry skin	Very frequent (80-99%)
HP:0001510	Growth delay	Very frequent (80-99%)
HP:0002231	Sparse body hair	Very frequent (80-99%)
HP:0002650	Scoliosis	Very frequent (80-99%)
HP:0004322	Short stature	Very frequent (80-99%)
HP:0005930	Abnormality of epiphysis morphology	Very frequent (80-99%)
HP:0008064	Ichthyosis	Very frequent (80-99%)
HP:0008905	Rhizomelia	Very frequent (80-99%)
HP:0009826	Limb undergrowth	Very frequent (80-99%)
HP:0010655	Epiphyseal stippling	Very frequent (80-99%)
HP:0001376	Limitation of joint mobility	Frequent (30-79%)
HP:0003298	Spina bifida occulta	Frequent (30-79%)
HP:0012368	Flat face	Frequent (30-79%)
HP:0001596	Alopecia	Occasional (5-29%)
HP:0010864	Intellectual disability, severe	Occasional (5-29%)

Identifiers

Disease identifiers

Field	Value
Canonical name	rhizomelic chondrodysplasia punctata
Mondo ID	MONDO:0015776
MeSH	D018902
OMIM	215100
Orphanet	177
DOID	DOID:2580
ICD-10-CM	E71.540
ICD-11	260357080
NCIT	C85047
SNOMED CT	56692003
UMLS	C0282529
MedGen	79471
GARD	0013160
Is cancer (heuristic)	no

Also known as: RCDP · rhizomelic chondrodysplasia punctata · rhizomelic chondrodysplasia punctata syndrome · rhizomelic dwarfism

Data availability: 130 ClinVar variants · 12 cell lines.

Disease family

An umbrella term covering 4 Mondo subtypes.

Classification path: disease › human disease › disease by body system or component › disorder of orbital region › eye disorder › rhizomelic chondrodysplasia punctata

Related subtypes (119): ptosis, eye accommodation disease, corneal disorder, asthenopia, lens disorder, keratomalacia, scleral disorder, ocular siderosis, coloboma, luxation of globe, mucopolysaccharidosis type 1, lacrimal apparatus disorder, Foster-Kennedy syndrome, anterior dislocation of lens, uveal disorder, eyelid disorder, ocular hypotension, scotoma, exophthalmos, ophthalmia nodosa, eye degenerative disorder, refractive error, glaucoma, retinal disorder, eye allergy, ocular vascular disorder, optic neuritis, conjunctival disorder, ocular hypertension, Tietz syndrome, Alagille syndrome, glaucoma-sleep apnea syndrome, Marshall syndrome, microcornea-glaucoma-absent frontal sinuses syndrome, nail-patella syndrome, oculodentodigital dysplasia, piebaldism, Sturge-Weber syndrome, cerebrotendinous xanthomatosis, ocular cystinosis, alpha-mannosidosis, megalocornea-intellectual disability syndrome, mucolipidosis type IV, mucopolysaccharidosis type 6, Netherton syndrome, galactosialidosis, Niemann-Pick disease type A, ocular motor apraxia, Cogan type, Peters plus syndrome, isolated Pierre-Robin syndrome, ectodermal dysplasia-blindness syndrome, Sandhoff disease, SHORT syndrome, Sjogren-Larsson syndrome, Smith-Lemli-Opitz syndrome, Tay-Sachs disease, tyrosinemia type II, Ito hypomelanosis, X-linked cone dysfunction syndrome with myopia, red color blindness, oculocerebrorenal syndrome, Lowry-MacLean syndrome, pigment dispersion syndrome, hereditary hyperferritinemia with congenital cataracts, dyssegmental dysplasia-glaucoma syndrome, mevalonic aciduria, familial cavitary optic disk anomaly, blindness - scoliosis - arachnodactyly syndrome, fatty acyl-CoA reductase 1 deficiency, microcephaly-intellectual disability-sensorineural hearing loss-epilepsy-abnormal muscle tone syndrome, neurotrophic keratopathy, Cogan syndrome, atopic keratoconjunctivitis, Ehlers-Danlos syndrome, kyphoscoliotic type 1, IRVAN syndrome, Rothmund-Thomson syndrome type 2, microcornea-corectopia-macular hypoplasia syndrome, isolated anophthalmia-microphthalmia syndrome, Spasmus nutans, toxic maculopathy due to antimalarial drugs, syndromic recessive X-linked ichthyosis, acute zonal occult outer retinopathy, acute annular outer retinopathy, phakomatosis pigmentovascularis, lamellar ichthyosis, idiopathic linear interstitial keratitis, chondroectodermal dysplasia with night blindness, galactosemia, GM1 gangliosidosis, Gaucher disease, visual snow syndrome, extensive peripapillary myelinated nerve fibers, IgG4-related ophthalmic disorder, global developmental delay-visual anomalies-progressive cerebellar atrophy-truncal hypotonia syndrome, vernal keratoconjunctivitis, Gardner syndrome, anterior segment dysgenesis, isolated ankyloblepharon filiforme adnatum, hereditary optic neuropathy, essential strabismus, Axenfeld anomaly, eye neoplasm, isolated blepharochalasis, punctate inner choroidopathy, eye infectious disorder, vitreous body disorder, 9q33.3q34.11 microdeletion syndrome, autoimmune/inflammatory optic neuropathy, LTBP2-related ocular dysgenesis, ocular growth disorder, ocular dysgenesis caused by defects in PAX6 regulation, choroidal neovascularization, anterior segment developmental abnormality with extraocular manifestations, congenital optic disk excavation, neuroocular syndrome, isolated angioid streaks, multiple evanescent white dot syndrome, stellate multiform amelanotic choroidopathy, macular telangiectasia

Subtypes (4): rhizomelic chondrodysplasia punctata type 1, rhizomelic chondrodysplasia punctata type 2, rhizomelic chondrodysplasia punctata type 3, peroxisome biogenesis disorder due to PEX5 defect in the PEX7-binding domain

Genetics & variants

GWAS landscape

No GWAS associations recorded — common-variant (GWAS) studies don’t cover this disease (typical for Mendelian / rare diseases). See the curated gene cohort and Mendelian overlap below.

Variant details and genetic-evidence tiers

ClinVar germline variants

130 retrieved; paginated sample, class counts are floors:

49 uncertain significance, 17 pathogenic/likely pathogenic, 17 likely benign, 17 likely pathogenic, 13 pathogenic, 7 benign, 6 conflicting classifications of pathogenicity, 4 benign/likely benign

ClinVar	Variant (HGVS)	Gene	Classification	Review
3366567	NC_000002.11:g.(178362494_178364345)_(178372760_178378546)del	AGPS	Pathogenic	criteria provided, single submitter
1073464	NM_014236.4(GNPAT):c.1583_1586del (p.Leu528fs)	GNPAT	Pathogenic	criteria provided, multiple submitters, no conflicts
190410	NM_001351132.2(PEX5):c.677dup (p.Val227fs)	PEX5	Pathogenic	criteria provided, single submitter
1075998	NM_000288.4(PEX7):c.474_477del (p.Tyr159fs)	PEX7	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
1339708	NM_000288.4(PEX7):c.376C>T (p.Gln126Ter)	PEX7	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
1421713	NM_000288.4(PEX7):c.517del (p.Ser173fs)	PEX7	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
1449189	NM_000288.4(PEX7):c.488G>A (p.Trp163Ter)	PEX7	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
1486959	NM_000288.4(PEX7):c.804-2A>C	PEX7	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
188711	NM_000288.4(PEX7):c.400G>A (p.Asp134Asn)	PEX7	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
188975	NM_000288.4(PEX7):c.188+1G>C	PEX7	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
189076	NM_000288.4(PEX7):c.618G>A (p.Trp206Ter)	PEX7	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
1926974	NM_000288.4(PEX7):c.858_867del (p.Glu287fs)	PEX7	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
225436	NM_000288.4(PEX7):c.183del (p.Phe61fs)	PEX7	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
3619127	NM_000288.4(PEX7):c.678G>A (p.Trp226Ter)	PEX7	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
370629	NM_000288.4(PEX7):c.13_19dup (p.Gly7fs)	PEX7	Pathogenic	criteria provided, multiple submitters, no conflicts
370682	NM_000288.4(PEX7):c.277C>T (p.Gln93Ter)	PEX7	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
4059703	NM_000288.4(PEX7):c.903+2T>G	PEX7	Pathogenic	criteria provided, single submitter
4818131	NM_000288.4(PEX7):c.804-2A>G	PEX7	Pathogenic	criteria provided, single submitter
4818134	NM_000288.4(PEX7):c.901_903+3delinsAG	PEX7	Pathogenic	criteria provided, single submitter
554785	NM_000288.4(PEX7):c.429del (p.Val144fs)	PEX7	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
639671	NM_000288.4(PEX7):c.130+1G>A	PEX7	Pathogenic	criteria provided, multiple submitters, no conflicts
7780	NM_000288.4(PEX7):c.875T>A (p.Leu292Ter)	PEX7	Pathogenic	criteria provided, multiple submitters, no conflicts
7781	NM_000288.4(PEX7):c.653C>T (p.Ala218Val)	PEX7	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
7782	NM_000288.4(PEX7):c.649G>A (p.Gly217Arg)	PEX7	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
7783	NM_000288.4(PEX7):c.694C>T (p.Arg232Ter)	PEX7	Pathogenic	criteria provided, multiple submitters, no conflicts
7785	NM_000288.4(PEX7):c.903+1G>C	PEX7	Pathogenic	criteria provided, multiple submitters, no conflicts
7786	NM_000288.4(PEX7):c.345T>G (p.Tyr115Ter)	PEX7	Pathogenic	criteria provided, multiple submitters, no conflicts
7788	NM_000288.4(PEX7):c.120C>G (p.Tyr40Ter)	PEX7	Pathogenic	criteria provided, multiple submitters, no conflicts
7790	NM_000288.4(PEX7):c.40A>C (p.Thr14Pro)	PEX7	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts
813363	NM_000288.4(PEX7):c.188+1G>A	PEX7	Pathogenic/Likely pathogenic	criteria provided, multiple submitters, no conflicts

Genes & proteins

Mendelian disease overlap and somatic drivers

GenCC: 0 · Orphanet: 8 · OMIM-shared: 0 · Dual-evidence (GWAS+Mendelian): 0

Orphanet rare-disease linkage (cohort genes)

Gene	Orphanet ID	Rare disease
AGPS	Orphanet:309803	Rhizomelic chondrodysplasia punctata type 3
GNPAT	Orphanet:309796	Rhizomelic chondrodysplasia punctata type 2
PEX7	Orphanet:309789	Rhizomelic chondrodysplasia punctata type 1
PEX7	Orphanet:773	Adult Refsum disease
PEX5	Orphanet:44	Neonatal adrenoleukodystrophy
PEX5	Orphanet:468717	Rhizomelic chondrodysplasia punctata type 5
PEX5	Orphanet:772	Infantile Refsum disease
PEX5	Orphanet:912	Zellweger syndrome

Cohort genes → proteins

4 cohort genes, 4 distinct canonical proteins.

Evidence partition

Subset	Genes
multi_evidence	4

Cohort genes (full)

Symbol	HGNC	Ensembl	UniProt	Name	Evidence
AGPS	HGNC:327	ENSG00000018510	O00116	Alkyldihydroxyacetonephosphate synthase, peroxisomal	clinvar
GNPAT	HGNC:4416	ENSG00000116906	O15228	Dihydroxyacetone phosphate acyltransferase	clinvar
PEX7	HGNC:8860	ENSG00000112357	O00628	Peroxisomal targeting signal 2 receptor	clinvar
PEX5	HGNC:9719	ENSG00000139197	P50542	Peroxisomal targeting signal 1 receptor	clinvar

Cohort function summary

Lead sentence per gene, UniProt-curated.

Symbol	Protein name	Function (lead sentence)
AGPS	Alkyldihydroxyacetonephosphate synthase, peroxisomal	Catalyzes the exchange of the acyl chain in acyl-dihydroxyacetonephosphate (acyl-DHAP) for a long chain fatty alcohol, yielding the first ether linked intermediate, i.e. alkyl-dihydroxyacetonephosphate (alkyl-DHAP), in the pathway of ether…
GNPAT	Dihydroxyacetone phosphate acyltransferase	Dihydroxyacetonephosphate acyltransferase catalyzing the first step in the biosynthesis of plasmalogens, a subset of phospholipids that differ from other glycerolipids by having an alkyl chain attached through a vinyl ether linkage at the…
PEX7	Peroxisomal targeting signal 2 receptor	Receptor required for the peroxisomal import of proteins containing a C-terminal PTS2-type peroxisomal targeting signal.
PEX5	Peroxisomal targeting signal 1 receptor	Receptor that mediates peroxisomal import of proteins containing a C-terminal PTS1-type tripeptide peroxisomal targeting signal (SKL-type).

Protein-family classification

Druggable: 2 · Difficult: 1 · Unknown: 1 · Druggable fraction: 0.5

Family distribution

Cohort families vs a genome-wide background (hypergeometric, BH-FDR; fold = observed/expected). Counts kept; sorted by enrichment, so the catch-all Other/Unknown bucket no longer leads.

Family	Genes	Fold	FDR
Enzyme (other)	2	6.0×	0.112
Scaffold/PPI	1	4.3×	0.318
Other/Unknown	1	0.5×	0.962

Per-gene assignment

Symbol	Family	Druggable?	EC	InterPro (top 3)
AGPS	Enzyme (other)	yes	2.5.1.26	FAD-bd_oxidored_4_C, Oxid_FAD_bind_N, FAD-linked_Oxase-like_C
GNPAT	Enzyme (other)	yes	2.3.1.42	Plipid/glycerol_acylTrfase, GPAT/DHAPAT, DHAPAT
PEX7	Scaffold/PPI	no		WD40_rpt, WD40/YVTN_repeat-like_dom_sf, WD40_repeat_CS
PEX5	Other/Unknown	no		TPR-like_helical_dom_sf, TPR_rpt, PEX5/PEX5L

Expression context

Cohort genes with no expression data: 0.

4 cohort genes are a single-cell marker in ≥1 SCXA experiment.

Breadth distribution (Bgee present_calls)

Bucket	Genes
narrow (1-5 tissues)	0
moderate (6-20)	0
broad (>20)	4
unknown	0

Top tissues across cohort

Tissue	Cohort genes
sperm	2
calcaneal tendon	1
tendon	1
biceps brachii	1
gluteal muscle	1
skeletal muscle tissue of biceps brachii	1
esophagus squamous epithelium	1
pigmented layer of retina	1
gastrocnemius	1
left testis	1
right testis	1

Per-gene tissue summary (top 30)

Symbol	Bgee breadth	FANTOM5 breadth	SCXA	Top tissues
AGPS	278	ubiquitous	marker	sperm, calcaneal tendon, tendon
GNPAT	294	ubiquitous	marker	gluteal muscle, biceps brachii, skeletal muscle tissue of biceps brachii
PEX7	276	ubiquitous	marker	pigmented layer of retina, sperm, esophagus squamous epithelium
PEX5	142	ubiquitous	marker	gastrocnemius, left testis, right testis

Protein interactions among cohort

Intra-cohort edges: 5.

Hub genes (top 10 by interactor count)

Symbol	Interactor count
AGPS	2,656
PEX5	1,741
GNPAT	1,641
PEX7	1,356

Intra-cohort edges

A	B	Sources
AGPS	GNPAT	string_interaction
AGPS	PEX5	string_interaction
AGPS	PEX7	string_interaction
GNPAT	PEX7	string_interaction
PEX5	PEX7	intact, string_interaction

Structural data

PDB: 1 · AlphaFold-only: 3 · No structure: 0

Cohort genes with PDB structures (top 30)

Symbol	UniProt	PDB entries
PEX5	P50542	11

AlphaFold-only cohort genes (top 30 by pLDDT)

Symbol	UniProt	pLDDT
PEX7	O00628	95.54
AGPS	O00116	89.27
GNPAT	O15228	88.96

Function

Pathway analysis

Distinct Reactome pathways touched by cohort: 10. Enrichment computed across 4 evidence-associated genes (4 with Reactome annotation).

Pathways by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 4 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

Pathway	Cohort genes	Fold	FDR	Sample cohort genes
Peroxisomal protein import	4	173.0×	1e-08	AGPS, GNPAT, PEX7, PEX5
Plasmalogen biosynthesis	2	1903.3×	1e-06	AGPS, GNPAT
Wax and plasmalogen biosynthesis	1	951.7×	0.004	AGPS
TYSND1 cleaves peroxisomal proteins	1	356.9×	0.007	AGPS
Pexophagy	1	237.9×	0.008	PEX5
Synthesis of PA	1	73.2×	0.023	GNPAT
E3 ubiquitin ligases ubiquitinate target proteins	1	48.4×	0.026	PEX5
Protein localization	1	47.6×	0.026	AGPS
Metabolism of lipids	1	7.9×	0.134	AGPS
Metabolism	1	2.9×	0.302	AGPS

GO biological processes by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 4 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

GO term	Cohort genes	Fold	FDR	Sample cohort genes
ether lipid biosynthetic process	3	1404.3×	1e-08	AGPS, GNPAT, PEX7
protein targeting to peroxisome	2	842.6×	3e-05	PEX7, PEX5
protein import into peroxisome matrix	2	702.2×	3e-05	PEX7, PEX5
fatty acid beta-oxidation	2	187.2×	3e-04	PEX7, PEX5
neuron migration	2	66.9×	0.002	PEX7, PEX5
cerebellum morphogenesis	1	1404.3×	0.004	GNPAT
protein import into peroxisome matrix, docking	1	1053.2×	0.004	PEX5
protein import into peroxisome matrix, translocation	1	1053.2×	0.004	PEX5
protein import into peroxisome matrix, substrate release	1	842.6×	0.004	PEX5
paranodal junction assembly	1	702.2×	0.004	GNPAT
protein import into peroxisome membrane	1	702.2×	0.004	PEX5
mitochondrial membrane organization	1	601.9×	0.004	PEX5
protein import into peroxisome matrix, receptor recycling	1	601.9×	0.004	PEX5
cerebral cortex cell migration	1	383.0×	0.006	PEX5
cerebral cortex neuron differentiation	1	300.9×	0.007	PEX5
pexophagy	1	263.3×	0.007	PEX5
lipid biosynthetic process	1	247.8×	0.007	AGPS
cell development	1	221.7×	0.008	PEX5
peroxisome organization	1	200.6×	0.008	PEX7
very long-chain fatty acid metabolic process	1	191.5×	0.008	PEX5
protein tetramerization	1	156.0×	0.009	PEX5
endochondral ossification	1	135.9×	0.009	PEX7
neuromuscular process	1	131.7×	0.009	PEX5
phosphatidic acid biosynthetic process	1	127.7×	0.009	GNPAT
membrane organization	1	127.7×	0.009	GNPAT
positive regulation of multicellular organism growth	1	123.9×	0.009	PEX5
negative regulation of protein-containing complex assembly	1	113.9×	0.010	PEX5
endoplasmic reticulum organization	1	105.3×	0.010	PEX5
cellular response to reactive oxygen species	1	102.8×	0.010	PEX5
synapse assembly	1	57.7×	0.017	GNPAT

Therapeutics

Drug target analysis

Approved (phase 4): 0 · Phase ≥3: 0 · Phased (≥1): 0 · Undrugged: 4

Druggability breadth: 2 of 4 evidence-associated genes (50%) have a ChEMBL target (buckets above are over the deeply-mined display cohort).

Top cohort targets by molecule count

Symbol	Molecules	Max phase
AGPS	0	0
GNPAT	0	0
PEX7	0	0
PEX5	0	0

Bioactivity and enzyme data

Enzyme cohort genes (≥1 EC): 2.

Cohort genes with ChEMBL bioactivity (full, sorted by assay count)

Symbol	Assays	Type breakdown
GNPAT	14	Functional:8, Binding:6
AGPS	5	Binding:5

Cohort enzymes (BRENDA EC)

Symbol	EC numbers	Names
AGPS	2.5.1.26	alkylglycerone-phosphate synthase
GNPAT	2.3.1.42	glycerone-phosphate O-acyltransferase

Pharmacogenomics

Cohort genes with a PharmGKB record: 4; with CPIC/DPWG dosing guidelines: 0.

No cohort gene has a CPIC/DPWG genotype-guided dosing guideline (PharmGKB).

Chemical tractability of cohort targets

0 approved/phased compounds have measured bioactivity against a cohort gene (and aren’t yet in disease-level trials). This is a research / tractability signal, NOT a therapeutic recommendation — a bioactivity row often reflects off-target or screening binding (e.g. promiscuous kinase inhibitors against a cohort kinase), implying no disease mechanism.

Druggability pyramid

Cohort genes binned by druggability tier (high → low):

Tier	Definition	Genes	Symbols
A	Approved (phase 4 drug)	0
B	Phased (≥1) drug, not yet approved	0
C	Druggable family + PDB, no drug	0
D	Druggable family + AlphaFold only, no drug	2	AGPS, GNPAT
E	Difficult family or no structure, no drug	2	PEX7, PEX5

Undrugged target profiles

4 cohort genes are undrugged. Ranked by ‘starting-point quality’ (assay depth + drugged-partner adjacency).

Symbol	ChEMBL assays	Drugged partners (top 3)
AGPS	5	—
GNPAT	14	—
PEX7	0	—
PEX5	0	—

Clinical trials & evidence

Clinical trials

Clinical trials: 2.

Phase distribution (across all retrieved trials)

Phase	Trials
PHASE1	1
Not specified	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT05969977	PHASE1	UNKNOWN	A First-in-Human Phase 1 Study of Plasmalogen Precursor PPI-1011 in Healthy Adult Volunteers to Assess Safety, Tolerability, and Pharmacokinetics
NCT04031287	Not specified	UNKNOWN	RCDP Natural History Study

Drugs tested across these trials (top 30)

Molecule	Max phase	Trials referencing
PPI-1011	1	1